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Patient Advocates Work Better if They Speak FDA's Language

Patient advocates of the rare disorder chronic fatigue syndrome (CFS) filed up to the podium during the public hearing portion of an FDA advisory committee meeting last month on a drug for the condition.

They went to voice their support for rintatolimod (Ampligen), when there is no other FDA-approved drug to treat CFS. The condition, which afflicts up to 4 million people, is defined as 6 or more consecutive months of profound fatigue that is not improved by bed rest and is worsened by mental or physical activity.

More than 30 patients and their family members pleaded for FDA approval in nearly 2 hours of testimony. Some FDA advisory committee meetings I've attended don't have a single patient advocate to speak in favor of the drug or device the agency is considering.

Here is where all those patient advocates failed: They based their pleas to the FDA on emotion rather than scientific data.

They told stories about how much a treatment was needed for CFS and offered anecdotes on how rintatolimod helped them regain some small amount of normalcy in their life.

Patients spoke about how CFS causes them to sleep their lives away and how the drug offers a sense of hope for a patient community struggling for help.

But the FDA doesn't base its decisions on emotion. It approves drugs whose benefits outweigh the risks, and rintatolimod had questionable evidence for both.

Those 30-some patient advocates didn't seem to care or understand that fact.

Their testimony was all for naught. The FDA advisory committee voted against recommending approval, and the agency is still reviewing the manufacturer's application.

Advisory committee members questioned the drug's efficacy and lack of long-term safety data presented to the FDA.

I can't help but think the patients would have had better results had they framed their arguments differently. If they understood the FDA's concerns about approving rintatolimod, they could have addressed those specifically.

Instead, their most common argument seemed to be, "We need a treatment option, this is all we have, so approve it."

They didn't recognize the questionable efficacy results and FDA's concerns about approving a drug with a shaky record.

Scientists work at the FDA, and patient advocates need to base their arguments on scientific reasoning rather than emotion.

Other disease groups are more organized and versed in FDA ways than the CFS one is, but everybody would stand a better chance of winning their way if patient groups understood the FDA's way of thinking and how the agency operates.

David Pittman is MedPage Today’s Washington Correspondent, following the intersection of policy and healthcare. He covers Congress, FDA, and other health agencies in Washington, as well as major healthcare events. David holds bachelors’ degrees in journalism and chemistry from the University of Georgia and previously worked at the Amarillo Globe-News in Texas, Chemical & Engineering News and most recently FDAnews.